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Rare Metabolic Disease That Leads to Childhood Obesity Gets Its First FDA-Approved Drug

MedCity News

Soleno Therapeutics Vykat XR is now FDA approved for treating hyperphagia, or excessive hunger, in patients with Prader-Willi syndrome. The once-daily pill is the first approved therapy for this rare disease, a leading cause of childhood obesity.

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GSK Multiple Myeloma Drug’s DREAMM Comeback Continues With Phase 3 Data at ASH

MedCity News

Results from the Phase 3 DREAMM-7 study show GSK drug Blenrep is helping multiple myeloma patients live longer compared to treatment with blockbuster Johnson & Johnson drug Darzalex. The post GSK Multiple Myeloma Drugs DREAMM Comeback Continues With Phase 3 Data at ASH appeared first on MedCity News.

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Theseus Pharma Lays Off Staff, Explores Options Months After Toxicity Scuttles Lead Drug

MedCity News

Theseus Pharmaceuticals discontinued its former lead program in July following the report of dose-limiting toxicities in two patients. Though Theseus has other assets in its pipeline, it has decided to explore strategic alternatives with the goal of “maximizing shareholder value.”

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Patient Death Leads to Pause in Test of 2seventy Bio Cell Therapy for Leukemia

MedCity News

2seventy Bio reported that an acute myeloid leukemia patient treated with its experimental cell therapy has died, prompting research partner Seattle Children’s Research Institute to pause the Phase 1 study. The FDA has been notified and an investigation is underway.

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FDA Reconsideration Leads to Approval of Takeda Drug for Rare Esophagus Disorder

MedCity News

FDA approval of Takeda Pharmaceutical drug Eohilia introduces a new therapeutic option for patients with eosinophilic esophagitis, inflammation of the esophagus that causes swallowing difficulty. It will compete against Dupixent, a Sanofi and Regeneron Pharmaceuticals drug already approved for this disorder.

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Cardiac Complication Leads FDA to Place Foghorn Cancer Drug Under Partial Hold

MedCity News

A clinical trial for a Foghorn Therapeutics cancer drug is under a partial FDA hold after a patient in the early-stage study developed a cardiovascular complication. It’s the biotech’s second partial hold in the past year.

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New Hemophilia Data Lead FDA to Delay Decision for BioMarin Gene Therapy

MedCity News

BioMarin Pharmaceutical’s submission of additional data for patients treated with its hemophilia A gene therapy, Roctavian, mean that the FDA will push out a regulatory decision to early summer. But it could be worth the wait as analysts anticipate FDA approval and project Roctavian becoming one of BioMarin’s biggest products.

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